FDA Grants Orphan Drug Designation to BEA-17 for Glioblastoma

The FDA has granted an orphan drug designation to BEA-17 for the treatment of patients with glioblastoma.



The FDA has granted an orphan drug designation to BEA-17 for the treatment of patients with glioblastoma, according to an announcement from Beactica Therapeutics.1

BEA-17 is a first-in-class, non-PROTAC small molecule targeted degrader of the epigenetic enzyme LSD1 and its co-factor CoREST. LSD1 overexpression is involved in the pathogenesis of a variety of cancers, including glioblastoma, for which there are currently no approved therapies that target this enzyme.

“The FDA’s decision to grant orphan drug status to BEA-17 marks an important milestone for the program and highlights the significant need for novel therapies to treat these devastating brain tumors,” Per Källblad, PhD, co-founder and chief executive officer of Beactica Therapeutics, said in a press release. “The designation will facilitate our development of this agent which we believe has the potential to serve as a much-needed therapeutic option for patients affected by glioblastoma.”

Glioblastoma is the most common and most aggressive brain tumor. Approximately 35,000 people in the United States and Europe will receive a diagnosis of glioblastoma every year, which is associated with a median overall survival (OS) of 15 months and a 5-year OS rate of only 5%.

Catalytic LSD1 inhibitors, although currently in clinical development, target only the catalytic binding site on the protein, resulting in limited anti-tumor activity.2 As such, researchers are investigating alternative approaches that can also disrupt the scaffolding function of LSD1.

In preclinical models, BEA-17 has shown in vivo potentiation of immune-modulating treatments in several cancer types, including standard-of-care temozolomide (Temodar) and radiation. Additionally, the compound has been shown to potentiate the activity of checkpoint inhibitors in syngeneic models of colon cancer. Neither effect has been proven with catalytic LSD1 inhibitors.

Moreover, pharmacokinetic studies of BEA-17 show evidence of blood-brain barrier penetration and oral availability.

In June 2019, the company announced that it had received a grant of approximately $300,000 from SweLife and Medtech4Health for its research evaluating allosteric modulators of the LSD1 enzyme in glioblastoma.3 The funded project, which is part of a larger collaboration with Uppsala University, the Akademiska Hospital in Uppsala and the SciLifeLab Drug Discovery and Development Platform, will evaluate the synergistic activity of BEA-17 in combination with currently approved anticancer treatments.

Currently, BEA-17 remains investigational and has not received approval anywhere globally, as its efficacy and safety in humans have not been established.1


  1. Beatica Therapeutics receives FDA orphan drug designation for BEA-17 for the treatment of glioblastoma. News release. Beactica Therapeutics. February 1, 2023. Accessed February 1, 2023. https://www.beactica.com/press/beactica-therapeutics-receives-fda-orphan-drug-designation-for-bea-17-for-the-treatment-of-glioblast
  2. Pipeline. Beactica Therapeutics. Accessed February 1, 2023. https://www.beactica.com/pipeline
  3. Beactica wins grant to develop combination therapy for glioblastoma. News release. Beactica Therapeutics. June 18, 2019. Accessed February 1, 2023. https://www.beactica.com/press/beactica-wins-grant-to-develop-combination-therapy-for-glioblastoma
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